Aparito collaborates with Garmin to Integrate Smartwatches into Clinical Trial Platform

September 19 2023, 09:00 BST

Aparito, a digital health technology company, and Garmin, a leading global provider of smartwatches, fitness trackers and digital health solutions, have ratified their collaboration to integrate Garmin smartwatches into Aparito’s Atom5™ clinical trial platform. The collaboration aims to capture real-world data and develop new digital endpoints and will be managed by the Garmin Health enterprise business solutions team.

The integration of Garmin smartwatches with the Aparito Atom5™ platform allows patients to perform clinical outcome assessments (COAs) at home such as the six-minute walk test (6MWT), and passively capture data such as sleep, heart rate and activity levels, making clinical trials more accessible to patients and providing richer data to clinicians utilizing an integration of the Garmin Health SDK.

Dr. Elin Haf Davies, Aparito CEO, said, “We collaborated with Garmin due to the integrity of their Health SDK and their collaborative culture. Working together to connect Garmin devices directly to our applications ensures we get access to raw data rather than relying on black box metrics and algorithms.”

“A strong network of technology collaborators is essential for us – especially in the research and clinical trials space. Together with Aparito, we are glad to provide a solution to researchers and clinicians for unique access to physiological data that is both digital and scalable,” said Dr. Leon Brudy, Garmin Health Business Development Manager. 

Ongoing collaborations between Aparito and Garmin are in place across many projects, including early-phase clinical trials, registry studies and digital biomarker development.

Join Aparito and Garmin Health at SCOPE Europe in Barcelona this October 17 and 18 where they will share their progress in digitising the 6-Minute Walk Test in conjunction with Dr. Joe Newman from Royal Papworth Hospital and the PHA UK patient group.

For more information on our collaborative projects, please visit this Garmin blog and this Aparito blog.

About Aparito

Aparito’s mission is to digitise clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.

Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to digitise clinical trials using novel eCOA and generate continuous real-world data. 

Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies. 

Discover more about how we digitise clinical trials on LinkedIn, Twitter and at aparito.com

Aparito logo

About Garmin Health

​​​​​​​Garmin Health provides custom enterprise business solutions that leverage Garmin’s extensive wearable portfolio and high-quality sensor data for applications in the corporate wellness, population health, and patient monitoring markets. The Garmin Health API allows third parties to ask customers to share their data in accordance with their specific privacy policies. 

As part of a global company that designs, manufactures and ships products worldwide, Garmin Health supports its customers’ commerce and logistics needs, allowing enterprises to scale with a single, trusted provider. For more information, email Garmin’s press team, connect on LinkedIn, or visit online at garmin.com/health.​​​​

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Not everything that can be counted counts: staying focused on what matters to patients

An update from Aparito CEO, Dr Elin Haf Davies.

Over the last three months, I have been privileged to co-author three papers.

Each one is different, but each one with the same central theme, the patient, and each one with the word patient in the title: 

Tanya Collin-Histed, Madeline Stoodley, Kathleen Beusterien, Deborah Elstein, Dena H. Jaffe, Shoshana Revel-Vilk, Elin Haf Davies on behalf of the International Gaucher Alliance(2023) A global neuronopathic Gaucher disease registry (GARDIAN): a patient-led initiative (IGA) Orphanet Journal of Rare Diseases volume 18, Article number: 195

Aiyegbusi, O.L., McMullan, C., Hughes, S.E., Davies, E.H., et al. Considerations for patient and public involvement and engagement in health research. TLC Study Group (2023) Nature Medicine 

Wicks, P., Wahlstrom-Edwards, L., Fillingham, S., Downing, A., Davies E.H (2003) So You Want to Build Your Disease’s First Online Patient Registry: An Educational Guide for Patient Organizations Based on US and European Experience The Patient – Patient-Centered Outcomes Research volume 16pages 183–199

My co-authors for the three papers are different collaborators, but all are focused on the importance of incorporating the patient’s voice into research and enabling the patient’s voice to be captured in a methodologically robust way.

Turning their voices into data. 

Over the two-plus decades that I’ve worked in clinical research, I’ve been notably aware that Not everything that can be counted counts and not everything that counts can be counted (as attributed to Albert Einstein).

From the first clinical trial I supported I realised that the endpoints used in clinical trials very rarely reflect what’s important to patients and families in their day to day.

That first clinical trial remains the driver for much of my work in neuronopathic Gaucher disease, and despite the challenges along the way, our paper in Orphanet describing the “A global neuronopathic Gaucher disease registry (GARDIAN)” and our efforts to develop disease-specific Patient Reported Outcomes and data-driven insight to guide better clinical trial designs in the future. 

Ultimately, insight and new knowledge are generated through data, and data is required to convince the regulators and reimbursement agencies that new medicines have the efficacy and effectiveness to be approved and reimbursed. 

While it’s the personal story that wins the hearts and minds via a headline story, the personal story is not what gets medicines approved: “Bring me data, not tears” is the well-known line reported to be told by an FDA to a mother of a child living with a rare life-threatening illness while reviewing a dossier for approval. 

Put another way, “In God we trust, all others must bring data”. 

Much has changed in the role of patients in the design and approval of medicines over the last two decades. Our recent blog with Neil Bertelsen reflects on the birth of modern patient advocacy back in the 1980s.  “That led to the FDA forming what is now their patient engagement policy, and it started with its HIV-specific patient council that advised the FDA and was later expanded to include more diseases”.

As Aparito rapidly approaches its ninth birthday it’s time to pause and review the data to reflect on where we’re at today. Are we still driving towards the original mission? Personally, there is pride that I have managed to maintain the patient central to all that I aspired to do with Aparito. 

Being awarded the Made WITH Patient Raising Star for my work in patient engagement was also an important external reflection of our ongoing achievements via the Patient Group Accelerator, which brings me immense satisfaction

Is what is being measured truly what matters?

But there seems to be a dichotomy between the achievements that can be measured in numbers and the aspects that bring me the most pride. 

How does one quantify the achievements over the last nine years? Do we focus on

  • the number of patients that have engaged with our Atom5™ platform?
  • the number of features that Atom5™ now has?
  • the number of studies that we’ve implemented?
  • the number of countries that we’re in?
  • the number of scientific publications and their respective impact factors?
  • the number of staff that we employ?
  • the amount of investment we’ve raised?
  • the amount of revenue growth that we can show year-on-year? 

It’s easy to be pulled away by other priorities; product, processes, pricing – all inevitable demands to be considered while working towards the ultimate goal of patient value.

But working on a product without considering the patient, focusing on the process without involving the patient, and negotiating a price without evaluating the patient value is to be distracted from the main purpose. 

What a decade it’s been to grow a global med-tech company: a pandemic, war in Europe, raging fires from the impact of climate change, and a high number of refugees losing their lives as they attempt to cross waters for a safer life. 

Some notable digital health companies that closed significant investments and sky-high valuations have crumbled, and the macro-economic continues to be unpredictable.  

As the pressures of building a company take their toll, I now have greater confidence to walk away from those who don’t buy into the mission, and to only work with people who have the same vision during the highs and the lows! 

People that stick with you through thick and thin, and are good, fun, kind people to work with.  

As we look towards our 10th year, I remain fully committed to the goal that I set for myself when I founded Aparito, to support patient-centric clinical trials by measuring what matters to patients. 


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Video-Based Assessments to Digitise COA: the story of SARAhome

The trend toward digitisation of clinical trials and the myriad benefits that partial or full decentralisation can offer has been proven to reduce costs, shorten the time to market for new therapies and improve ROI.

The questions of digitisation’s impact on drug development are arguably answered, and whilst this has obvious benefits to sponsors, there remain questions regarding the validation and regulatory acceptance of digital endpoints and novel electronic Clinical Outcome Assessments (eCOAs).

In this white paper, we explore our journey to digitise an existing COA and the steps we took to develop, validate, and deploy SARAhome into real-world studies to measure the impact of ataxia on patients’ everyday lives, an approach that enabled us to develop an eCOA where daily or twice daily assessments provide a more meaningful measure of the severity of ataxia than a conventional snapshot visit conducted once a year.


Our white paper examines the past, present and future of ataxia assessments

The past

sara past

Since 2004, the clinical scale “Scale of Assessment and Rating of Ataxia (SARA)” presented by Schmitz-Hübsch T et al, 2006 has been used to measure and rate the severity of Ataxia. SARA remains an “in-hospital tool” with poor visibility of the daily fluctuation of patients’ conditions due to the infrequency of hospital visits.

The present

nose to finger sarahome

SARAhome uses a video-based assessment delivered via Atom5TM in the patient’s home with no limitation on the frequency of assessments that can be performed which can be reviewed by clinicians via the central assessor platform.

The future

pose estimation vtug

Aparito has demonstrated the feasibility and utility of applying pose-estimation analysis to video-recorded tasks and established the need for standardisation to generate the best results, which will continue to generate newer, more accurate digital endpoints.

Download the whitepaper here!

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New decentralised study to validate home-based video assessment of Duchenne muscular dystrophy announced

On World Duchenne Awareness Day, Duchenne UK and Aparito announce a new study to help validate a new approach, the DMDhome digital platform, to testing drugs in clinical trials.

The DMDhome platform is the result of a successful collaboration between Duchenne UK and Aparito which uses video capture and computer vision analysis to measure limb function in Duchenne muscular dystrophy (DMD). The study will aim to validate this new approach for use in clinical trials and potentially in the approval of new medicines for DMD.

Run by health technology company Aparito using the Atom5™ clinical trial platform and co-funded by Duchenne UK and Joining Jack, the study will focus on assessing upper and lower limb function in DMD, particularly in the transfer phase which is the transition from the ambulant (walking) to non-ambulant (non-walking) stage.

The study will be fully decentralised which means that those taking part can do so in their home as opposed to having to travel to a clinical site.

DMDhome – a new electronic clinical outcome assessment

DMDhome was developed following Duchenne UK being chosen to join Aparito’s Patient Group Accelerator Programme in 2020 with the intention of devising a patient-centred electronic clinical outcome assessment (eCOA), which was able to capture changes in functional ability and their impact on meaningful daily-life activities.

The aim of the video capture and computer vision assessment was to identify disease features specific to DMD and the best way to capture and measure them.

This initial work found that the DMDhome platform is a viable way to give and record instructions for motor tasks to participants via video and to collect the results that participants report, including quality of life data capture using the DMD Quality of Life Measure (DMD QoL).

The proof of concept data was published in 2022.

Aims of DMD validation study

A rigorous clinical validation approach to test DMDhome will be rolled out in a natural history study (a study that tracks the course of a disease over time) to assess the clinical significance and reliability of new electronic clinical outcome assessments (eCOAs) compared to the traditional assessments currently used. As in the previous study, quality of life data from participants and their families will also be captured, using the DMD QoL.

It is expected that the study will lead to a comprehensive data set to support the use of DMDhome video assessments as digital endpoints in DMD clinical trials to assess both disease progression and potential drug benefit, and ultimately, in time, support its qualification as a eCOAs by medicines regulators.

Importance of study

Emphasising the importance of this work and its significance for people with DMD and their families, Alessandra Gaeta, Director of Research at Duchenne UK, said:

“The DMD ‘transfer stage’, which sees the transition from the ambulant to the non-ambulant stage of the disease, involves a huge change in quality of life for people living with the condition and their carers.  Despite this, there is no validated assessment in the clinic yet that captures this stage accurately and consistently.

“That’s why, once validated, DMDhome has the potential to become an important tool in the toolkit to assess and measure changes in motor function at this stage, supporting the understanding of impact of new treatments on the disease and on quality of life.”

The study has been designed by Aparito in collaboration with the University of Oxford. Laurent Servais, Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, who is the Principal Investigator, outlines its aims:

It is very exciting to take part in new developments in the field of digital outcomes. We need more granularity when we assess our patients – in order to build shorter and more efficient clinical trials. I am very happy to see the focus on a population that we neglect too often in outcome development and thus in clinical trial – the population of patients in transition

Laurent Servais, Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre

This study represents the consolidation of the partnership between Aparito and Duchenne UK and a great achievement for Aparito’s Patient Group Accelerator Programme, which supports the co-creation process of new eCOAs and digital endpoints with patients. Validating DMDhome will be a major step towards the acceptance and use of patient-centric digital outcome measures in rare disease medicine development.

Dr Elisa Ferrer Mallol, Patient Engagement Manager at Aparito

Recruitment for DMD Home validation study

The study will open recruitment of eligible participants over the next few months. This will be announced via the DMD Hub.


Facts and figures about DMD

What is DMD?
DMD is a genetic disease that is typically diagnosed in childhood between the age of three and six. It causes muscle weakness and wasting. It eventually affects all the muscles in the body, including the heart and lungs.
What causes DMD?

It is caused by a fault, known as a mutation, on the dystrophin gene. Dystrophin is a protein that protects muscles; without it, muscles are easily damaged, and their strength and function is weakened.
Prevalence
DMD is the most common and severe form of muscular dystrophy.DMD almost always affects boys and can affect any ethnicity. There are 2,500 people in the UK living with DMD.
Treatment 

There is currently no cure for Duchenne, but there are treatments and therapies that can slow down DMD progression and improve quality of life.
Current assessments 
Routine care and clinical trials frequently use the following clinical outcome assessment to evaluate disease progression: the 6MWT (six-minute walk test), NSAA (North Star Ambulatory Assessment) and PUL (Performance of Upper Limb scale).

About Aparito
Aparito’s mission is to digitize clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments. Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to deliver digital clinical trials using novel eCOA to generate continuous real-world data. Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

Aparito logo

About Duchenne UK

Duchenne UK is a charity that was set up in 2012 by Emily Reuben and Alex Johnson following both of their sons being diagnosed with DMD. They set up Duchenne UK to tackle some of the big challenges in drug development in their search to accelerate the development of treatments. In 11 years, Duchenne UK has raised more than £20 million and used this money to:   

  • Fund clinical trials of medicines that are now showing promise for DMD 
  • Set up DMD Care UK, a national care programme for patients with DMD to stop patients with DMD from dying too young because they were not getting the right care. The programme establishes best practice across all the disciplines involved in DMD care and works to ensure all medical professionals and parents know precisely what treatment children and adults with DMD need. 
  • Create a DMD medical research hub with hospital sites across the country, which has led to more trials for DMD treatments than ever before.  
  • Develop innovative technologies, the SMART Suit and Dream Chair, to support the independence of people with DMD.  

You can find out more about the work of Duchenne UK at duchenneuk.org

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SARAhome video assessment deployed in pan-European ataxia observational study

The Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app paper is the latest development in our work with DZNE and is available to read via the BMJ.

PROFA will see 200 patients enrolled in a six-month validation and observational study and will recruit from six centres across Germany, France, and Austria utilising the SARAhome assessment via our Atom5™ clinical trial platform.

Introduction

Friedreich ataxia (FA) is the most common hereditary ataxia in Europe, characterised by progressively worsening movement and speech impairments with a typical onset before the age of 25 years. The symptoms affect the patients’ health-related quality of life (HRQoL) and psychosocial health.

FA leads to an increasing need for care, associated with an economic burden. Little is known about the impact of FA on daily lives and HRQoL.

To fill that gap, we will assess patient-reported, psychosocial and economic outcomes using momentary data assessment via a mobile health application (app).

Methods and analysis 

The PROFA Study is a prospective observational study. Patients with FA (n=200) will be recruited at six European study centres (Germany, France and Austria). We will interview patients at baseline in the study centre and subsequently assess the patients’ health at home via mobile health app. Patients will self-report ataxia severity, HRQoL, speech and hearing disabilities, coping strategies and well-being, health services usage, adverse health events and productivity losses due to informal care on a daily to monthly basis on the app for 6 months. Our study aims to (1) validate measurements of HRQoL and psychosocial health, (2) assess the usability of the mobile health app, and (3) use descriptive and multivariate statistics to analyse patient-reported and economic outcomes and the interaction effects between these outcomes. Insights into the app’s usability could be used for future studies using momentary data assessments to measure outcomes of patients with FA.


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Accelerometer-derived sleep measures in idiopathic dystonia

In Accelerometer-derived sleep measures in idiopathic dystonia: A UK Biobank cohort study, the study demonstrates the utility of accelerometers in longer term evaluation of sleep in dystonia, for measurement of disturbance and response to treatment. Compared to controls, altered sleep and circadian rhythm were more common in dystonia patients which may contribute to the clinical phenotype.

Accelerometer-derived sleep measures in idiopathic dystonia wearable devices

Background

Sleep disturbance is an increasingly recognized non-motor trait in dystonia, with varying findings reported to date. Here, we examine sleep in a UK Biobank-derived dystonia cohort using subjective self-reported sleep symptoms and objective accelerometer-derived sleep measures, with comparison to a control population.

Methods

A total of 241 dystonia cases were compared to 964 matched controls in analysis of self-reported sleep symptoms and changes in sleep architecture using wrist-worn triaxial accelerometers.

Results

Dystonia participants had poorer self-reported sleep patterns compared to controls. Accelerometery measurements demonstrated later sleep times, reduced time in bed, and shifts in circadian rhythm. No association was observed with pain, and only limited relationships with psychiatric symptoms.


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First patient recruited for the EJP-DT4RD European Study at the Institute of Myology

August 09 2023, 09:00 CEST

Since announcing the launch of the Digital Tools for Rare Disease project (DT4RD), a product of the Rare Disease Research call placed by the European Joint Programme on Rare Diseases in partnership with Fondation Maladies Rares sponsored by Chiesi and CSL Behring, extensive progress has been made!

Non-invasive tools for measuring rare disease patient mobility in daily living and distinguishing between voluntary and involuntary movements were put in place using state-of-the-art wearable sensors integrated with Aparito’s Atom5TM clinical trial platform to capture physiological and psychosocial parameters. 

The main objective of this project has been focused on developing and validating a way to acquire, transfer, store and process heterogeneous data related to movement measured at home and anchored to assessments performed at the hospital, as Clinical Outcome Measures (eCOA).

This European study involves great collaboration with renowned clinical experts located at the Institute of Myology (Paris, France), and at the John Walton Muscular Dystrophy Research Centre (Newcastle, UK).

After the French Ethics Committee’s approval, the Institute of Myology is proud to announce their First Patient recruited into the study on the 3rd of July 2023!

Welcoming the first patient to the DT4RD study on July 3 2023

The enrolment went very well although a bit longer than expected to explain in detail the protocol and train the patient with the digitised tools including the Atom5TM platform with video tasks and ePRO to complete, the Garmin Vivosmart5 wearable device and the Yumen Bionics wearable sensors.

Dr Jean-Yves Hogrel, Director of the Laboratory of Neuromuscular Physiology and Evaluation at the Institute of Myology

The patient expressed her motivation for taking part in this study related to the mobility of patients affected by rare neuromuscular diseases. Afterwards, she declared: “I felt comfortable, not too tired and confident to carry the tasks at home independently”.

The Institute of Myology Team added: “We are in the process of recruiting very soon our next patient on the 1st of September 2023 and keep on enrolling a total of 20 patients in the next few months.”

This is a great milestone which reflects a wonderful collaboration to date. We are always grateful when patients enrol on our studies and appreciate their time and dedication to develop innovation that will hopefully be used in patient-centric clinical trials to convey what’s important to patients.

Dr Elin Haf Davies, CEO of Aparito

By digitising critical procedures within the clinical trial process, Aparito leads this project with European Clinical partners as well as renowned technology and industry partners to provide real-time visibility of data collection and integrity, reducing patient and site burden, enhancing the patient experience, and improving patient retention.

This EJP-DT4RD study is supported by the Atom5TM platform’s innovative technology in the patient’s homes using disease-specific smartphone apps, video assessments, and wearable technology to deliver meaningful data through eCOAs and the development of digital biomarkers.


About Aparito
Aparito’s mission is to digitize clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments. Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to deliver digital clinical trials using novel eCOA to generate continuous real-world data. Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

Aparito logo

About the Institute of Myology

The Institute of Myology, located in Paris, coordinates medical management, research, and education related to muscle diseases and injuries. It is an international reference centre that participates in numerous trials and clinical studies, with a focus on neuromuscular diseases, high-performance sports, and ageing. The Institute of Myology Association facilitates the coordination of site activities in partnership with five public guardianship organisations, and the institute includes a complete Center of Research in Myology and several scientific poles as well as clinical activities.

Find them on https://www.institut-myologie.org/en/

Institut de Myologie logo

About the EJP-DT4RD project

A unique project involving Academics (Institute of Myology, John Walton Muscular Dystrophy Research Centre), SMEs (Aparito, Yumen Bionics), large pharmaceutical companies (Chiesi, CSL Behring), and a patient organisation (MSUK) working towards the same objective for and with patient involvement. It widens the horizon of tremendous possibilities for the future of research in rare diseases and warrants hope for future clinical trials using Real World data.

Find out more at https://www.ejprarediseases.org/rare-diseases-research-rdr-challenge-1-digital-tools-for-rare-disease-dt4rd/

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Provisioned Devices vs BYOD in Clinical Trials

The last decade has seen a paradigm shift towards hybrid trials and DCTs. This transformation reached its peak during the COVID-19 pandemic with unprecedented challenges faced by the clinical community, including interrupted trials and potential impact on the lives of millions of patients.

The choice between a PD or BYOD in clinical trials is based on the protocol data capture needs, sponsor preference, patient needs, and risk mitigation. Ethics committee approvals can also influence the decision. However, when it comes to the quality of data – a core requirement in a clinical trial – evidence is growing for there being no difference.

The challenges that the sponsor must assess while deciding whether to use PDs or BYOD are diverse.

We share what we have learnt, along with our clients, about the benefits and risks of using PD or BYOD for sponsors, sites, and patients.


Our whitepaper sets out several findings including

The past

sara past

Since 2004, the clinical scale “Scale of Assessment and Rating of Ataxia (SARA)” presented by Schmitz-Hübsch T et al, 2006 has been used to measure and rate the severity of Ataxia. SARA remains an “in-hospital tool” with poor visibility of the daily fluctuation of patients’ conditions due to the infrequency of hospital visits.

The present

nose to finger sarahome

SARAhome uses a video-based assessment delivered via Atom5TM in the patient’s home with no limitation on the frequency of assessments that can be performed which can be reviewed by clinicians via the central assessor platform.

The future

pose estimation vtug

Aparito has demonstrated the feasibility and utility of applying pose-estimation analysis to video-recorded tasks and established the need for standardisation to generate the best results, which will continue to generate newer, more accurate digital endpoints.

Download the whitepaper here!

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Aparito and TruLab Partner to Improve Rare Disease Studies

July 31 2023, 09:00 EST

Aparito and TruLab, two companies focused on improving rare disease studies, have formed a partnership to improve the efficiency and experience of clinical trials.

Aparito and TruLab logos

TruLab’s technology has gained traction in the rare disease space due to the difficulty of obtaining samples, especially from children, and the criticality of the sample for the patient’s eligibility in the study. Both Aparito’s and TruLab’s mobile platforms are designed to maintain data integrity and minimize the potential for data loss.

By leveraging Aparito’s Atom5™ platform, clinical trials can be supported in the patients’ homes using disease-specific smartphone apps, video assessments, and wearable technology to deliver meaningful data through eCOAs and digital biomarkers.

Collectively the companies intend to reduce the burden on parents and caregivers seeking treatment for their children. The application of innovative technologies in this setting ensures that patient samples and data points are collected according to study procedures and are not lost, ultimately increasing the chances of success in the clinical trial.

By digitizing critical procedures within the clinical trial process, TruLab and Aparito can provide real-time visibility to data collection and integrity, reduce patient and site burden, enhance the patient experience, and improve patient retention.

“We are excited to partner with TruLab to improve the collection and management of samples in rare disease studies,” said Dr Elin Haf Davies, CEO of Aparito. “Our Atom5™ platform will help ensure that the patient-generated data collected is of the highest quality, ultimately leading to a better clinical trial experience for people living with rare diseases.”

“We are delighted to partner with Aparito to provide a seamless solution for rare disease studies,” said Jim Wahl, Vice President of Client Services at TruLab. “Our combined technologies will enable us to provide high-quality data to support the development of treatments for rare diseases.”

The partnership between Aparito and TruLab is a significant step forward in rare disease research. With the two companies working together, researchers will have access to platforms that provide real-time visibility and improve the quality of data collected and ultimately lead to better treatments for patients with rare diseases.

About Aparito
Aparito’s mission is to digitize clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.

Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to digitise clinical trials using novel eCOA and generate continuous real-world data. 

Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

Discover more at aparito.com.

Aparito logo

About TruLab
TruLab, Inc. is a sample management software company that provides advanced point-of-collection workflow solutions for the clinical research industry.

TruLab’s technology is designed to help clinicians, labs and pharmaceutical sponsors manage their clinical samples more effectively, while ensuring consistent compliance with study protocols and achieving target levels of data quantity and quality.

TruLab also enables real-time visibility of clinical procedures and sample status for pharmaceutical and lab customers, especially in a decentralized trial environment.

Learn more at trulab.com.

TruLab Logo

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A global neuronopathic Gaucher disease registry (GaRDIAN): a patient-led initiative

We talk often about breaking new ground in rare disease but GaRDIAN represents a true watershed moment in patient empowerment.

GaRDIAN is a patient-led, patient-owned registry for neuronopathic Gaucher disease (nGD), a rare condition that has a significant impact on patients and caregivers, and where there is minimal natural history combined with small patient numbers and huge heterogeneity within the same genotype.

“We are delighted that our manuscript on GaRDIAN has been published,” said Tanya Collin-Histed, Chief Executive Officer of the International Gaucher Alliance (IGA), “and we believe GaRDIAN will be an important tool in the future for improving care and developing safer and more targeted medicines.”

A global neuronopathic gaucher disease registry (GaRDIAN): a patient-led initiative

 

Background

Gaucher disease (GD) is a rare autosomal recessive lysosomal storage disorder. GD types 2 and 3 are known as neuronopathic Gaucher disease (nGD) because they have brain involvement that progresses over time. Implementing a systematic approach to the collection of real-world clinical and patient-relevant outcomes data in nGD presents an opportunity to fill critical knowledge gaps and ultimately help healthcare providers in the management of this patient population. This paper summarizes the development of a patient-initiated Gaucher Registry for Development Innovation and Analysis of Neuronopathic Disease (GARDIAN).

GaRDIAN was the brainchild of the International Gaucher Alliance and Elin Haf Davies of Aparito,” says Tanya, “an ally in our community for over two decades. We hope that by sharing our experience of how we developed GaRDIAN and the disease-specific patient-reported and observer-reported outcomes for nGD we will validate in the registry, and other patient communities will be encouraged to lead the way in their disease areas.”

GaRDIAN intends to bridge the many gaps in the understanding of nGD and align with regulatory frameworks on real-world data needs.


Access the paper

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